A pill to stop the bleeding? new hope for HHT nosebleeds
NCT ID NCT07743671
First seen Aug 04, 2026 · Last updated Sep 18, 2026 · Updated 5 times
Summary
This phase 3 trial tests whether an experimental oral drug called engasertib can reduce the number of nosebleeds in adults with moderate to severe hereditary hemorrhagic telangiectasia (HHT), a genetic condition that causes abnormal blood vessels and frequent bleeding. About 240 participants will receive either engasertib or a placebo daily for 28 weeks. The main goal is to see if the drug lowers the total number of nosebleed episodes compared to placebo.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- engasertib (also called VAD044), taken as an oral capsule
- What this could lead to
- If it works, engasertib could become the first approved treatment to reduce frequent nosebleeds in people with HHT, improving daily life and reducing the need for transfusions.
- What could go wrong
- This is a phase 3 trial, but success is not guaranteed. The drug may not reduce nosebleeds more than a placebo, and side effects are possible. Results will need to be confirmed in this larger study.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 3
Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.
- Participants
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About 240 people
The number the study aims to enrol. It can still change while the study runs.
- Expected to start
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Sep 2026
An estimate. Start dates often move.
- Expected to finish
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Jun 2028
An estimate. End dates often move.
- Lead sponsor
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A company
The lead sponsor is a pharmaceutical, biotech, or medical-device company.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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18 years and older
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: 1. Participants are ≥18 years of age at the Screening Visit. 2. Participants have a definite diagnosis of HHT by the Curaçao criteria, defined as spontaneous and recurrent epistaxis and having at least 2 of the following criteria: 1. Multiple telangiectases at characteristic sites: lips, oral cavity, fingers, or nose; 2. Visceral lesions: gastrointestinal telangiectasia and/or pulmonary, hepatic, cerebral, or spinal arteriovenous malformations (AVMs); or 3. A first degree relative with HHT according to these criteria. 3. Participants must have an ESS \>4 at screening, and, in the judgement of the Investigator, participants are expected to have regular epistaxis that typically lasts for several minutes. This criterion is assessed at screening only and does not require reconfirmation prior to randomization on Day 1. 4. Participants have anemia OR in the prior 6 months have received a parenteral infusion of at least 250 mg of iron OR in the prior 6 months have received a red cell or whole blood transfusion. Exclusion Criteria: 1. History or current diagnosis of clinically significant electrocardiogram (ECG) abnormalities. 2. History of significant or uncontrolled skin disorders per Investigator's judgement. 3. Local ablative (eg, cauterization) or surgical procedures on nasal telangiectases \<6 weeks before the Screening Visit. 4. Use of drugs with anti-angiogenic properties, including, but not limited to, bevacizumab, pazopanib, thalidomide, lenalidomide, pomalidomide, tacrolimus, sirolimus, or selective estrogen response modulators (tamoxifen, raloxifene, or bazedoxifene) \< 6 weeks before the Screening Visit. 5. Use of oral tranexamic or epsilon-aminocaproic acid unless they are on a stable dose for at least 4 weeks before the Screening Visit, which will need to be continued during the entire duration of the double-blind Treatment Period.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The study's own enquiry address
This study publishes an address for enquiries. See it below .
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The places running it
4 sites. The list below names each one and where it is.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Show contact details
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Genom att skicka in godkänner du våra Användarvillkor
Study contacts
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Contact
Email: •••••@•••••
Locations
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Innovative Hematology, Inc.,
RECRUITINGIndianapolis, Indiana, 46260, United States
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Mayo Clinic
RECRUITINGRochester, Minnesota, 55905, United States
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The University of Chicago Medical Center (UCMC)
RECRUITINGChicago, Illinois, 60637, United States
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Washington University School of Medicine
RECRUITINGSt Louis, Missouri, 63110, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
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- Massive HHT registry aims to unlock secrets of rare bleeding disorder
- Blood thinners in HHT: a risky balancing act under the microscope
- Can a transplant drug stop HHT nosebleeds? small study tests sirolimus
- New antibody aims to tame rare bleeding disorder
- New drug aims to stop nosebleeds in rare blood vessel disorder